Severe sickle cell disease-pathophys- iology and therapy. Biol Blood Marrow Transplant. 2010;16(1Suppl):S64-S67.Buchanan, G., Vichinskym E., Krishnamurti, L. & Shalini, S. (2011) Severe sickle cell disease - pathophysiology and therapy. Blood Marrow Transplant 16(1 Suppl): S64-S67....
-Exa-cel PDUFA target action date is December 8, 2023 for severe sickle cell disease (SCD)- ZUG, Switzerland and BOSTON, Oct. 31, 2023 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) today announced the completion of the U.S. Food and Drug Administration’s (FDA)...
Sickle Cell DiseaseClinical Management of Severe Sickle Cell Disease Yardumian A. · Davies S.C.Department of Haematology, Central Middlesex Hospital, London, UK Acta Haematol 1987;78:193–197 (DOI:10.1159/000205874)Abstract Patients affected by sickle cell disease encounter varied and often severe...
“This means individuals with sickle cell disease who come to the hospital presenting with pain should also be tested for Covid-19,” said Lana Mucalo, from the Medical College of Wisconsin. Children living with SCD who had previously suffered more than two pain events requiring acute care...
The Sickle Cell Unrelated Donor Transplant Trial (SCURT trial) of the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) is a phase II study of the toxicity and efficacy of unrelated donor hematopoietic cell transplantation in children with severe sickle cell disease (SCD) using a ...
The Singh-led study assessed COVID-19 outcomes among 122 people with sickle cell disease, comparing them to a group of more than 15,000 Black people who don't have sickle cell but do have other health problems that make them vulnerable to severe COVID-19. Those other risk factors include...
Durable Clinical Benefits with Exagamglogene Autotemcel for Severe Sickle Cell Diseasedoi:10.1182/blood-2024-204001Frangoul:Vertex Pharmaceuticals:Consultancy;... H Frangoul,F Locatelli,A Sharma,... - 《Blood》 被引量: 0发表: 2024年 5612617EFFICACY AND SAFETY OF A SINGLE DOSE OF EXAGAMGLOGEN...
Stem Cell Transplant for Severe Sickle Cell Myeloablative allogeneic hematopoietic stem cell transplantation (HSCT) is curative for children with severe sickle cell disease, but toxicity may be prohi... Matthew M Hsieh,Courtney D Fitzhugh,R Patrick Weitzel,... - 《Jama》 被引量: 90发表: 2014年...
Voxelotor Treatment of a Patient With Sickle Cell Disease and Very Severe AnemiaThe case involves a 38‐year‐old female of Nigerian descent with sickle cell anemia (SCA, βS/βS genotype), no α‐globin gene deletion and no glucose 6‐phosphate dehydrogenase deficiency. Over the past two ...
Noun1.severe combined immunodeficiency disease- a congenital disease affecting T cells that can result from a mutation in any one of several different genes; children with it are susceptible to infectious disease; if untreated it is lethal within the first year or two of life ...